Cyclophosphamide in Treating Young Patients With Severe Autoimmune Enteropathy

NCT00258180 | Completed Phase 2 Results

• 5 other identifiers: 03-07-08-04P30CA006973JHOC-J0326J0326CDR0000441133
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

RATIONALE: Cyclophosphamide may help control the symptoms of autoimmune enteropathy . PURPOSE: This phase II trial is studying how well cyclophosphamide works in treating young patients with severe autoimmune enteropathy.

Conditions

Diarrhea; Gastrointestinal Complications; Unspecified Childhood Solid Tumor, Protocol Specific

Keywords

unspecified childhood solid tumor, protocol specific; gastrointestinal complications; diarrhea

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-free Remission at 1 Year After Study Completion

  Number of participants off therapy 1 year after study completion without relapse.

  1 year

• Number of Participants Experiencing Intervention-related Adverse Events, as Defined by CTCAE at 1 Month

  1 month

Study Arms (1)

severe autoimmune enteropathy

EXPERIMENTAL

Young patients with severe autoimmune enteropathy receive cyclophosphamide IV over 1 hour on days 1-4. Patients then receive filgrastim (G-CSF) IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover

Biological: filgrastim; Drug: cyclophosphamide

Interventions

filgrastim BIOLOGICAL

Administered IV or subcutaneously once daily beginning on day 10 and continuing for 3 days or until blood counts recover

Also known as: G-CSF

severe autoimmune enteropathy

cyclophosphamide DRUG

Administered IV over 1 hour on days 1-4

severe autoimmune enteropathy

Eligibility Criteria

• Age: 1 Year - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

DISEASE CHARACTERISTICS: * Diagnosis of severe autoimmune enteropathy * Condition is resistant to conventional therapy * Histologic evidence of severe villous atrophy with intense lymphocytic infiltrate of the lamina propria by small intestinal biopsy within the past 3 months * Disease failed to respond after ≥ 2 months of corticosteroid therapy at a dose of ≥ 0.5 mg/kg/day or ≥ 40 mg/day for patients \> 20 kg AND 1 of the following therapies: * Cyclosporine resulting in ≥ 1 whole blood level of \> 200 ng/mL * Tacrolimus resulting in ≥ 1 whole blood level of 5 ng/mL * At least 50% estimated caloric needs provided by parenteral nutrition * History of intractable diarrhea, defined as frequent watery stools for \> 3 months that does not respond to dietary restriction * No celiac disease, defined by a history of positive antiendomysial antibody or tissue transglutaminase antibody * No primary immunodeficiency or x-linked autoimmunity-allergy dysregulation PATIENT CHARACTERISTICS: Performance status * Lansky 60-100% Life expectancy * Not specified Hematopoietic * Not specified Hepatic * Not specified Renal * Not specified Cardiovascular * Ejection fraction ≥ 40% OR shortening fraction ≥ 20% Pulmonary * FVC or FEV\_1 ≥ 50% of predicted (for patients \> 8 years of age) * No clinically abnormal pulmonary function or abnormal pulse oximetry (for patients ≤ 8 years of age) Other * Not pregnant * Negative pregnancy test * Fertile patients must use effective contraception during and for at least 9 months after completion of study treatment * No known chromosomal abnormality PRIOR CONCURRENT THERAPY: Biologic therapy * No immunizations for at least 6 months after completion of study treatment Endocrine therapy * See Disease Characteristics * At least 5 days since prior corticosteroids * No concurrent dexamethasone as an anti-emetic Other * At least 5 days since other prior immunosuppressive medications (e.g., tacrolimus or cyclosporine)

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Johns Hopkins University: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21231-2410, United States

Location

MeSH Terms

Conditions

Diarrhea

Interventions

Filgrastim; Granulocyte Colony-Stimulating Factor; Cyclophosphamide

Condition Hierarchy (Ancestors)

Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Results Point of Contact

• Title: Maria Oliva-Hemker MD
• Organization: Johns Hopkins School of Medicine

moliva@jhmi.edu

410-955-8769

Study Officials

• David M. Loeb, MD, PhD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  PRINCIPAL INVESTIGATOR

• Maria Oliva-Hemker, MD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 22, 2005
• First Posted: November 24, 2005
• Study Start: August 15, 2005
• Primary Completion: February 24, 2009
• Study Completion: February 24, 2009
• Last Updated: April 16, 2019
• Results First Posted: April 16, 2019

Record last verified: 2019-03

Locations

US (1)